An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

Hunter Syndrome Clinical Trial

Official title:

A Multi-Center Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients Enrolled in the Hunter Outcome Survey (HOS) Receiving Idursulfase Enzyme Replacement Therapy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00882921
• Other study ID #: HGT-ELA-042
• Status: Completed
• Start date: October 14, 2008
• Est. completion date: February 8, 2013

Study information

• Verified date: May 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The objective of this study is to evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do not after long-term idursulfase enzyme replacement therapy (ERT).

Description:

This study is being conducted to satisfy post-marketing commitments to monitor anti-idursulfase antibody development in Hunter syndrome patients after long-term idursulfase enzyme replacement therapy. The study will be conducted as a sub-study within the Hunter Outcome Survey (HOS). Hunter syndrome patients in the HOS who have previously received idursulfase as well as treatment-naive patients who will begin idursulfase treatment within 30 days of study enrollment will be included.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 26
• Est. completion date: February 8, 2013
• Est. primary completion date: February 8, 2013
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years and older

Eligibility

Inclusion Criteria:

Patients must meet all of the following criteria to be considered eligible for enrollment:

• The patient is male and enrolled in the HOS (i.e., meets the entry criteria of a documented diagnosis of Hunter syndrome)

• The patient is = 5 years-old

• The patient is on idursulfase treatment or scheduled to begin idursulfase treatment within 30 days of study enrollment

• The patient, patient's parent(s), or patient's legally authorized guardian must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient, patient's parent(s), or patient's legally authorized guardian.

Exclusion Criteria:

Patients who meet any of the following criteria are not eligible for this study:

• The patient has received biologic/ERT products other than idursulfase, or other investigational product(s) for any reason within 30 days prior to study entry.

• The patient has a life expectancy of < 2 years

• The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult; has an uncooperative attitude; is unable to return for safety evaluations; or is otherwise unlikely to complete the study, as determined by the Investigator.

Related Conditions & MeSH terms

• Hunter Syndrome
• Mucopolysaccharidosis II
• Syndrome

Intervention

Biological:
• Idursulfase
Patients received idursulfase as prescribed by their physician following locally approved prescribing information. Patients will not be provided idursulfase by Shire Human Genetic Therapies, Inc. or the HOS.

Locations

Country	Name	City	State
Brazil	Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica	Porto Alegre	RS
United Kingdom	Birmingham Children's Hospital	Birmingham
United Kingdom	Great Ormond Street Hospital	London
United Kingdom	Central Manchester University Hospitals, St. Mary's Hospital	Manchester
United States	Children's Hospitals and Clinics of Minnesota, Division of Genetics	Minneapolis	Minnesota
United States	Children's Hospital & Research Center Oakland	Oakland	California

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Countries where clinical trial is conducted

United States,  Brazil,  United Kingdom, 

References & Publications (1)

Giugliani R, Harmatz P, Jones SA, Mendelsohn NJ, Vellodi A, Qiu Y, Hendriksz CJ, Vijayaraghavan S, Whiteman DA, Pano A. Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Infusion-Related Adverse Event (IRAE) Rates Between IgG Anti-idursulfase Antibody Positive (Ab+) and Anti-idursulfase IgG Antibody Negative (Ab-) Patients	The primary analysis of how presence of antibodies affected IRAE rates was performed based on a negative binomial regression model. This was done to account for potentially differential follow-up time between antibody groups.	Baseline to 109 Weeks
Secondary	Change From Baseline in uGAG Levels to 109 Weeks	Urine GAG	Baseline to 109 Weeks